[November 24, 2014]

CureDuchenne Media Statement Regarding BioMarin's and Prosensa Holding N.V.'s Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Muscular Dystrophy Products to Rare Disease Portfolio

CureDuchenne is encouraged about BioMarin Pharmaceutical's acquisition of Prosensa and their commitment to continue the development of drugs for Duchenne muscular dystrophy. BioMarin has a deep understanding of the orphan rare disease space, experience working with regulatory authorities worldwide, and a successful record of getting drugs approved. CureDuchenne supports all research efforts that further the development of treatment options for boys with Duchenne.

CureDuchenne has played a critical role in helping to fund Prosensa for more tha 10 years that has helped bring drisapersen to its current stage of development. Drisapersen is currently under a rolling review as part of the New Drug Application process. CureDuchenne has also funded Prosensa's other exon skipping compounds that are under development.

"CureDuchenne has been a dedicated supporter of Prosensa since our company's inception," said Hans Schikan, Chief Executive Officer of Prosensa. "CureDuchenne's funding came at critical times during our drug development process and their longtime commitment has been of enormous value to the company. The transaction with BioMarin will enhance our mission by bringing innovative therapies to patients across the world as quickly as possible."

"Our commitment to finding treatments for Duchenne is unwavering," said Debra Miller, Founder and CEO of CureDuchenne. "We have been there every step of the way for Prosensa and are encouraged that BioMarin will continue this effort that will hopefully lead to approval of drisapersen and a pipeline of other follow on exons."

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